such as mutated BRCA genes. Germline mutations in the TP53 gene are uncommon and associated with a specific cancer syndrome known as Li-Fraumeni syndrome. People with Li-Fraumeni syndrome often ...
Access to genetic testing brings with it several advantages – one being early diagnosis for young patients with potentially life-threatening multi-system conditions. Retinal-renal syndrome ...
For instance, candidate Huntington’s drugs that aim to reduce activity of the mutated gene (or the protein it codes for) have struggled in clinical trials. The new study implies that because very few ...
Scientists have found 697 genetic variants – or small changes within a gene – linked to depression, of which almost 300 were previously unknown. A Queensland scientist described the project as ...
With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now found a commercialization partner for both the U.S. and Asian markets.
Many animals survived even after undergoing radiation-based genetic mutations—notably, a number of pet dogs abandoned by their owners during Chernobyl’s hasty evacuation orders. Today ...
The company is submitting the Hunter syndrome treatment for U.S. approval on a rolling basis, a process it expects to complete this quarter. An approval would make RGX-121 the first gene therapy for ...
That could change if not enough people get their seasonal flu shot. The potential for danger lies in H5N1’s genetic pliability, according to Edwin Michael, PhD, an epidemiologist at the ...
Abuhelwa Z, Ali NHA, Xie Z, et al. The argument for targeted therapy in IDH1 mutated myelodysplastic syndromes (MDS): poor outcomes post-hypomethylating agent failure in higher risk MDS and ...
The first human trial of insulin-producing cells that have been gene-edited to evade immune attack is a success so far. The cells have survived and produced insulin for a month after being ...
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